Cytokinetics reports topline results from SEQUOIA-HCM Phase 3 trial

Cytokinetics announced topline results from SEQUOIA-HCM, the pivotal Phase 3 clinical trial of aficamten in patients with symptomatic obstructive hypertrophic cardiomyopathy. The results of SEQUOIA-HCM show that treatment with aficamten significantly improved exercise capacity compared to placebo, increasing peak oxygen uptake measured by cardiopulmonary exercise testing by a least square mean difference of 1.74 mL/kg/min. The treatment effect with aficamten was consistent across all prespecified subgroups reflective of patient baseline characteristics and treatment strategies, including patients receiving or not receiving background beta-blocker therapy. Statistically significant and clinically meaningful improvements were also observed in all 10 prespecified secondary endpoints, including Kansas City Cardiomyopathy Questionnaire Clinical Summary Score at weeks 12 and 24, the proportion of patients with greater than or equal to1 class improvement in New York Heart Association functional class at weeks 12 and 24, change in provoked left ventricular outflow tract gradient and proportion less than30 mmHg at weeks 12 and 24, as well as exercise workload and guideline-eligibility for septal reduction therapy. Aficamten was well-tolerated in SEQUOIA-HCM with an adverse event profile comparable to placebo. Treatment emergent serious adverse events occurred in 8 and 13 patients on aficamten and placebo, respectively. Core echocardiographic left ventricular ejection fraction was observed to be less than50% in 5 patients on aficamten compared to 1 patient on placebo. There were no instances of worsening heart failure or treatment interruptions due to low LVEF.